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CNS Research Scientist

Spark Therapeutics
Philadelphia, Pennsylvania
Salary not provided
Closing date
Dec 20, 2021

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Health Sciences, Drug Development
Job Type
Research Scientist
Organization Type
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Job Details

Primary Duties

The Research Scientist is a member of the CNS Therapeutic Area within the Translational Research Department. He/she will be responsible for:

  • Design and construction of optimized AAV gene therapy vectors to drive therapeutic transgene expression derived from neuronal tissues.
  • Experimental design, execution and data analysis for CNS-directed research programs.
  • Ability to assume independent ownership of scientific programs.
  • Write study reports, manuscripts and present results at appropriate scientific meetings.
  • Demonstrated ability to independently initiate and oversee joint programs with internal and external collaborators.


% of Time Job Function and Description


Identify and validate new indication targets and optimize gene expression cassettes to support both existing and new programs in the pipeline. This includes executing experiments on the bench, extensive literature review, championing product candidate development from proof of concept to IND submission, etc.


Prepare sample analysis reports, present results to various internal teams, and write parts of regulatory submissions to regulatory agencies.

Education and Experience Requirements

  • Typically has a PhD in a scientific discipline and 3 years of related experience. May also have a Bachelor’s Degree or Master’s degree and 3 years of related experience.
  • Hands-on research experience, ideally with gene therapies targeting the CNS
  • Familiarity with target lead identification/optimization using a combination of molecular, cellular and in vitro/in vivo techniques.
  • Experience with studies in laboratory animal models
  • Experience with AAV vectors and CNS gene therapy is preferred, but not required

Key Skills, Abilities, and Competencies

  • Experience in general and advanced lab techniques such as molecular cloning and plasmid design and construction, qPCR, ELISA, cell culture, plate-based luciferase and/or enzymatic activity assays, SDS-PAGE/western blotting, etc.
  • Experience with studies in laboratory animals, including stereotaxic surgery and behavioral studies.
  • Familiarity with IND enabling studies, regulatory filings, and GLP studies is a plus
  • Strong analytical skills and problem-solving capabilities
  • Proven ability to work independently in hands-on laboratory setting, self-motivated to learn and develop new techniques, perform multiple tasks simultaneously, keep accurate records, follow instructions, and comply with company policies.
  • Excellent communication skills (both oral and written)
  • Familiar with computer software commonly used in bio/pharma industries.
  • Works with colleagues as a team player.
  • Strong organizational skills.
  • Strong publication record is a plus.

Please be aware that Spark mandates COVID-19 vaccination of all employees regardless of work location. Accommodations may be made in accordance with applicable law.

Nearest Major Market: Philadelphia

Join the Spark Team

We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our shared mission is to challenge the inevitability of genetic disease by discovering, developing, and delivering treatments in ways unimaginable - until now.

We don't follow footsteps. We create the path.


At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.

Founded in March 2013 as a result of the technology and know-how accumulated over two decades at Children’s Hospital of Philadelphia (CHOP), our investigational therapies have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. Greater understanding of the human genome and genetic abnormalities have allowed our scientists to tailor investigational therapies to patients suffering from very specific genetic diseases. This approach holds great promise in developing effective treatments to a host of inherited diseases. Our initial focus is on treating orphan diseases.

Headquartered in dynamic Philadelphia, we are a diverse, experienced team united in our goal to break down barriers for people and families affected by genetic diseases. As our name suggests, our investigational, one-time therapies are designed to spark healthy biology, and deliver potentially life-altering transformation for people and families affected by genetic disease. Spark is a member of the Roche Group.

Find Us
3737 Market Street
Suite 1300
United States
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